2. PROJECT SUMMARY ? ADMINISTRATIVE CORE The Administrative Core will be responsible for the overall administration of Consortium operations. Oregon Health & Science University will house the coordinating center for the consortium. The Administrative Core will oversee the finances of the consortium, establish all policies and procedures of the consortium, maintain communication between the clinical research sites in the consortium and our advocacy group partner the NPKUA, and maintain communication with the Rare Disease Clinical Research Network (RDCRN) and the Office of Rare Diseases Research (ORDR) at the National Institute of Child Health and Human Development (NICHD). The Administrative Core will be responsible for the management of data between the consortium sites to the Data Management Coordinating Center (DMCC) and will also be responsible for the study design and biostatistical analysis of the data. Core personnel will be responsible for regulatory compliance across the consortium including preparation and submission of IRB protocols and development of Data Safety Monitoring Plans.